FDA Approves Nonsteroidal Drug for Duchenne

The FDA has approved Duvyzat (givinostat) to treat patients with Duchenne muscular dystrophy (DMD) who are six years of age and older.

Duchenne is a rare genetic disease that is characterized by a mutation in the dystrophin gene. It causes the muscles in the body to become weak and damaged over time and is eventually fatal. Duchenne affects about 1 in 3,500 to 5,000 boys born worldwide

Duvyzat is the first nonsteroidal oral drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and loss of muscle. Duvyzat was developed by Milan-based Italfarmaco in partnership with Telethon and Duchenne Parent Project (Italy). It will be marketed in the United States by ITF Therapeutics, a new subsidiary of Italfarmaco.

Company officials said they anticipate Duvyzat will be available in the third quarter of 2024. Dosing will be based on weight. The pricing has not yet been determined.

ITF Therapeutics will offer a range of services to support access to Duvzyat, Matt Trudeau, general manager of ITF Therapeutics, told Formulary Watch. “We are in the process of gathering feedback from the DMD community to ensure the programs and services we develop meet the needs of patients and their families,” he said.

Craig M. McDonald, M.D.

“There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. Duvyzat’s unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD,” Craig M. McDonald, M.D., professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health and a principal investigator, said in a press release.

The approval is based on the results of the pivotal phase 3 EPIDYS trial, which enrolled 179 boys six years of age or older who received either Duvyzat twice daily or placebo, in addition to glucocorticosteroid treatment. The study met its primary endpoint demonstrating that patients on Duvyzat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment.

Duvyzat also showed favorable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), a scale commonly used to rate the motor function in boys with DMD who are capable of walking.

The most common side effects of Duvyzat are diarrhea, abdominal pain, a decrease in platelets — which can lead to increased bleeding — nausea/vomiting, an increase in triglycerides (a type of fat in the body) and fever. The majority of adverse effects were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024.

The prescribing information includes warnings about evaluating patients’ platelet counts and triglycerides before prescribing Duvyzat. Additionally, Duvyzat may also cause QTc prolongation, which can increase the risk for irregular heartbeats.